MYOZYME 50 mg Порошок Для Инъекционного Раствора

Содержание инструкции

1. Myozyme 50 mg powder for concentrate for solution for infusion
   1. What is Myozyme and what is it used for
   2. What you need to know before you take Myozyme
   3. How to take Myozyme
   4. Possible side effects
   5. How to store Myozyme
   6. Contents of the pack and other information

Myozyme 50 mg powder for concentrate for solution for infusion

Alglucosidase alfa

Read this leaflet carefully before you use this medicine because it contains important
information for you.

• Keep this leaflet. You may need to read it again.
• If you have any questions, ask your doctor, pharmacist or nurse.
• If any side effects occur, including those not listed in this leaflet, ask your doctor, pharmacist or nurse. See section 4.

Contents of this leaflet:

• 1. What Myozyme is and what it is used for
• 2. What you need to know before you take Myozyme
• 3. How to take Myozyme
• 4. Possible side effects
• 5. How to store Myozyme
• 6. Contents of the pack and other information

1. What is Myozyme and what is it used for

Myozyme is used to treat adults, children and adolescents of any age with a confirmed diagnosis of Pompe disease.
People affected by Pompe disease have reduced levels of an enzyme called alpha-glucosidase.
This enzyme helps the body control glycogen levels (a type of carbohydrate).
Glycogen provides energy to the body, but in Pompe disease, glycogen levels can become too high.
Myozyme contains an artificial enzyme called alglucosidase alfa, which is able to replace the naturally deficient enzyme in Pompe disease.

2. What you need to know before you take Myozyme

Do not use Myozyme
If you have had previous life-threatening allergic reactions (hypersensitivity) to alglucosidase alfa or to any of the other ingredients of this medicine (listed in section 6) and if re-administration of the medicine was not effective. Symptoms of life-threatening allergic reactions include, among others, low blood pressure, very rapid heart rate, difficulty breathing, vomiting, swelling of the face, hives or rash.

Warnings and precautions
During treatment with Myozyme, you may experience an infusion-associated reaction during administration of the medicine or in the hours following the infusion. This reaction manifests with various symptoms such as low blood pressure, chest discomfort, tightness of the throat, swelling of the face, lips or tongue (angioedema), hives, dizziness, rash, itching, nausea, vomiting, cough and bronchospasm (see section 4 for an overview of all infusion-associated reactions). An infusion-associated reaction can be very severe in some cases. If you should experience such a reaction, contact your doctor immediately. You may need to receive medicines before treatment to prevent an allergic reaction (such as antihistamines and/or corticosteroids) or reduce fever (antipyretics).
In studies, doctors have used medicines to suppress the immune system and reduce antibody production. Since you have Pompe disease, there is a risk of developing a serious airway or lung infection. The use of these medicines to suppress the immune system could further increase this risk.
If you notice severe ulcerative skin lesions, inform your doctor. If you have swelling in the lower limbs or generalized edema, inform your doctor. The doctor must consider interrupting the administration of Myozyme and initiating appropriate medical treatment. The doctor must consider the risks and benefits of re-administering Myozyme.

Other medicines and Myozyme
Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines.

Pregnancy, breastfeeding and fertility
There is limited experience with the use of Myozyme in pregnant women. Myozyme should not be administered during pregnancy unless it is clearly necessary. Tell your doctor if you are breastfeeding. Limited experience suggests that Myozyme passes into breast milk in very small amounts. No effects are expected in breastfed infants. Therefore, breastfeeding during treatment can be considered. However, you may discuss with your doctor whether to interrupt breastfeeding as a precautionary measure for the first 24 hours after each dose of Myozyme. If you are pregnant, suspect you are pregnant, are planning to become pregnant, or are breastfeeding, ask your doctor or pharmacist for advice before taking this medicine.

Driving and operating machinery
Do not drive and do not use tools or machinery immediately after the Myozyme infusion, as dizziness, drowsiness, tremor and/or low blood pressure may occur.

Myozyme contains sodium
This medicine contains less than 1 mmol of sodium (23 mg) per vial, i.e. it is practically ‘sodium-free’.

3. How to take Myozyme

The administration of Myozyme must occur exclusively under the supervision of a physician
competent in the treatment of Pompe disease.
The administered dose is based on body weight. The recommended dose of Myozyme is 20 mg/kg
of body weight, administered once every 2 weeks.
Home infusion
The physician may deem that You can undergo home infusion of Myozyme if it is safe
and convenient to do so. If you experience side effects during a Myozyme infusion, the home
infusion staff may stop the infusion and initiate appropriate medical treatment.

Use in children and adolescents
The recommended dose of Myozyme in children and adolescents is the same as that of adults.

Instructions for correct use
Myozyme is administered via an intravenous line (intravenous infusion). It
comes as a powder to be mixed with sterile water before administration.

If you take more Myozyme than you should
If you are administered Myozyme at a dose or infusion rate higher than that
recommended, you may experience infusion-associated reactions. This reaction may include
symptoms such as:

• skin and lips that become blue due to lack of oxygen in the body tissues, increased heart rate, palpitations
• difficulty breathing, cough
• dizziness, headache, taste disturbances
• high blood pressure, hot flashes
• swelling of the tongue, vomiting, diarrhea, nausea
• chest pain, chest discomfort, throat tightness, fever, chills, feeling cold, redness at the infusion site
• muscle pain
• skin redness

If you experience such a reaction, inform your doctor immediately (see section 2). The
infusion rate will be reduced or the infusion will be stopped and, as appropriate, you may receive corrective
treatment.

If you forget to take Myozyme
If an infusion is missed, contact your doctor.
If you have any questions about the use of this medicine, ask your doctor, pharmacist or
nurse.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.
In patients, side effects have occurred mainly during or shortly after administration (“infusion-related
reactions”). Some of the infusion-related side effects were severe or potentially life-threatening. In
some patients, life-threatening reactions have been reported, including severe generalized allergic
reactions and anaphylactic shock. The symptoms of such reactions include low blood pressure, very fast
heart rate, difficulty breathing, vomiting, swelling of the face, lips or tongue, hives or skin rash.
Some patients have experienced infusion-related side effects in the form of flu-like symptoms, which
lasted for a few days after the infusion was completed.
If you notice any of these types of side effects, contact your doctor immediately. You may need
to receive medicines before treatment to prevent an allergic reaction (such as antihistamines and/or
corticosteroids) or to reduce fever (antipyretics).

Very common: may affect more than 1 in 10 people

• Hives
• Skin rash
• Increased heart rate
• Flushing (of the face)
• Fever or increased body temperature
• Cough
• Increased breathing rate
• Vomiting
• Reduced oxygen levels in the blood

Common: may affect up to 1 in 10 people

• Pallor
• Increased blood pressure/hypertension
• Bluish discoloration of the skin
• Chills
• Agitation
• Tremor
• Headache
• Tingling
• Pain or reaction at the infusion site
• Dizziness
• Irritability
• Itching
• Nausea
• Swelling of the face, swelling of the throat or severe swelling of the face, throat and tongue due to a
  severe allergic reaction
• Swelling of the arms and legs
• Nausea
• Chest discomfort
• Throat tightness
• Diarrhea
• Fatigue
• Muscle pain
• Muscle spasms
• Severe ulcerative skin lesions
• Skin redness

Not known:(frequency cannot be estimated from available data)

• Swelling around the eyes
• Asthma
• Abnormal sounds during breathing, including wheezing
• Difficulty breathing (including shortness of breath)
• Cold extremities (hands, feet)
• Hypotension (low or reduced blood pressure)
• Constriction of blood vessels causing decreased blood flow
• Sudden constriction of the airways, resulting in less air passage (bronchospasm)
• Feeling of warmth
• Feeling of cold
• General feeling of discomfort (malaise)
• Feeling of weakness
• Drowsiness
• Fainting
• Burning sensation
• Increased sweating
• Watery eyes
• Skin patches
• Restlessness
• Wheezing
• Throat irritation
• Lack of oxygen in the body tissues
• Decreased heart rate
• Cardiac arrest
• Strong heartbeat that may be fast or irregular (palpitations)
• Chest pain (not at the level of the heart)
• Inflammation of the membrane covering the eyes or eyelids
• Abdominal pain
• Indigestion
• Difficulty swallowing
• Joint pain
• Temporary interruption or sudden cessation of breathing
• Proteinuria
• Nephrotic syndrome: swelling of the lower limbs, generalized edema and proteinuria
• Swelling and thickening of the skin at the infusion site in case of leakage of the product from the blood
  vessels
• Redness of the palms
• Transient skin color change
• Redness at the infusion site
• Hives (skin rash) at the infusion site
• Itching at the infusion site
• Blisters

Reporting of side effects
If you get any side effects, talk to your doctor, pharmacist or nurse. This includes any side effects not
listed in this leaflet. You can also report side effects directly through the national reporting system
listed in Annex V.
By reporting side effects you can help provide more information on the safety of this medicine.

5. How to store Myozyme

Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date which is stated on the label after ‘Exp.’. The expiry
date refers to the last day of that month.
Store in a refrigerator (2 ºC – 8 ºC).
After dilution, it is recommended to use the product immediately. However, chemical and physical
stability has been demonstrated for 24 hours at 2 ºC – 8 ºC, if the medicine is protected from light.
Do not dispose of any medicine in wastewater or household waste. Ask your pharmacist how to
dispose of medicines you no longer use. This will help protect the environment.

6. Contents of the pack and other information

What Myozyme contains

• The active substance is alglucosidase alfa. One vial contains 50 mg of alglucosidase alfa. After reconstitution, the solution contains 5 mg of alglucosidase alfa per mL and following dilution the concentration ranges from 0.5 mg to 4 mg/mL.
• The excipients are
• mannitol (E421)
• disodium hydrogen phosphate monohydrate (E339)
• disodium phosphate heptahydrate (E339)
• polysorbate 80 (E433)

Description of the appearance of Myozyme and contents of the pack
Myozyme is a powder for concentrate for solution for infusion in a vial (50 mg/vial).
Each pack contains 1, 10 or 25 vials. Not all pack sizes may be
marketed.
The powder is from white to off-white in colour. After reconstitution, the solution is clear, from
colourless to pale yellow, and may contain particles. The reconstituted solution must be further
diluted.

Marketing Authorisation Holder and Manufacturer
Marketing Authorisation Holder
Sanofi B.V.
Paasheuvelweg 25
1105 BP Amsterdam
The Netherlands

Manufacturer
Genzyme Ireland Limited, IDA Industrial Park, Old Kilmeaden Road, Waterford, Ireland
For further information on this medicinal product, contact the local representative of the Marketing
Authorisation Holder:

België/Belgique/Belgien/
Luxembourg/Luxemburg
Sanofi Belgium
Tél/Tel: + 32 2 710 54 00

Magyarország
SANOFI-AVENTIS Zrt.
Tel: +36 1 505 0050

България
Swixx Biopharma EOOD
Тел.: +359 (0)2 4942 480

Malta
Sanofi S.r.l.
Tel: +39 02 39394275

Česká republika
Sanofi s.r.o.
Tel: +420 233086 111

Nederland
Sanofi B.V.
Tel: +31 20 245 4000

Danmark
Sanofi A/S
Tlf: +45 45 16 70 00

Norge
sanofi-aventis Norge AS
Tlf: + 47 67 10 71 00

Deutschland
Sanofi-Aventis Deutschland GmbH
Tel. 0800 04 36 996
Tel. aus dem Ausland: +49 69 305 70 13

Österreich
sanofi-aventis GmbH
Tel: + 43 1 80 185 – 0

Polska
Sanofi Sp. z o.o.
Tel.: +48 22 280 00 00

Ελλάδα
Sanofi-Aventis Μονοπρόσωπη AEBE
Τηλ: +30 210 900 1600

Eesti
Swixx Biopharma OÜ
Tel: +372 640 10 30

Portugal
Sanofi – Produtos Farmacêuticos, Lda.
Tel: +351 21 35 89 400

España
sanofi-aventis, S.A.
Tel: +34 93 485 94 00

România
Sanofi Romania SRL
Tel: +40 (0) 21 317 31 36

France
Sanofi Winthrop Industrie
Tél: 0 800 222 555
Appel depuis l’étranger: +33 1 57 63 23 23

Slovenija
Swixx Biopharma d.o.o.
Tel: +386 1 235 51 00

Hrvatska
Swixx Biopharma d.o.o.
Tel: +385 1 2078 500

Slovenská republika
Swixx Biopharma s.r.o.
Tel: +421 2 208 33 600

Ireland
Sanofi-aventis Iralenad Ltd. T/A SANOFI
Tel:+353(0)1 4035600

Ísland
Vistor hf.
Sími: +354 535 7000

Suomi/Finland
Sanofi Oy
Puh/Tel: + 358 201 200 300

Sverige
Sanofi AB
Tel: +46 (0)8 634 50 00

Italia
Sanofi S.r.l.
Tel: 800536389

United Kingdom (Northern Ireland)
sanofi-aventis Ireland Ltd. T/A SANOFI
Tel: +44 (0) 800 035 2525

Latvija
Swixx Biopharma SIA
Tel: +371 6 616 47 50

Κύπρος
C.A. Papaellinas Ltd.
Τηλ: +357 22 741741

Lietuva
Swixx Biopharma UAB
Tel: +370 5 236 91 40
More detailed information on this medicinal product is available on the website of the European
Medicines Agency: http://www.ema.europa.eu. Furthermore, links to other websites on rare diseases
and related therapeutic treatments are provided.
------------------------------------------------------------------------------------------------------------------>
The following information is intended exclusively for healthcare professionals:

Instructions for use – reconstitution, dilution and administration
Myozyme must be reconstituted with water for injections, then diluted with a
solution for injection of 9 mg/mL (0.9 %) sodium chloride, and then administered by
intravenous infusion. Reconstitution and dilution must be carried out in accordance with good
practice, in particular with regard to aseptic technique.
Due to the protein nature of the product, particles may form in the reconstituted solution and in the final
infusion bags. Therefore, use an in-line filter with low protein binding of 0.2 micron for administration. The use of a 0.2 micron in-line filter
has been shown to remove visible particles and does not cause any apparent loss of protein or activity.
Determine the number of vials to be reconstituted, based on the dosage regimen for the
individual patient (mg/kg), then remove the necessary number of vials from the refrigerator, to
reach room temperature (approximately 30 minutes). Each vial of Myozyme is for single use only.

Follow an aseptic technique
Reconstitution

• Reconstitute each 50 mg vial of Myozyme with 10.3 mL of water for injections using a syringe with a needle diameter of less than 20 gauge. Add the water for injections slowly and dropwise along the wall of the vial, and not directly onto the solid lyophilised medicinal product. Then tilt and gently rotate each vial, without inverting it, rotate it quickly or shake it. The reconstituted volume is 10.5 mL containing 5 mg of enzyme/mL and appears as a clear solution, from colourless to pale yellow, which may contain particles in the form of fine white filaments or translucent fibres. Immediately inspect the reconstituted vials if particulate matter and discolouration are noted. If inspection reveals foreign particles other than those described above, or if the solution is discoloured, do not use the product. The pH of the reconstituted solution is approximately 6.2.
• It is recommended to dilute the vials promptly after reconstitution (see below).

Dilution

• If the medicinal product is reconstituted as described above, the reconstituted solution inside the vial will contain 5 mg of alglucosidase alfa per mL. The reconstituted volume allows for the accurate aspiration of 10.0 mL (equivalent to 50 mg) from each vial. Subsequently, further dilute as follows: slowly aspirate the reconstituted solution from each vial, up to a volume equal to the dose for the patient using a syringe with a needle diameter of less than 20 gauge. The recommended final concentration of alglucosidase in the infusion bags should be from 0.5 mg/mL to 4 mg/mL. Remove air from the bags and also remove an equivalent volume of 9 mg/mL (0.9 %) sodium chloride solution for injection, to be replaced with reconstituted Myozyme. Slowly inject the reconstituted Myozyme directly into the 9 mg/mL (0.9 %) sodium chloride solution for injection. Invert and gently swirl the infusion bag to mix the diluted solution. Do not shake or vigorously agitate the infusion bag.
• Administer the final infusion solution as soon as possible.
• Unused medicinal product or waste material should be disposed of in accordance with local requirements.

Administration
It is recommended to start administering the diluted solution within three hours. The total time from
reconstitution to the end of the infusion should not exceed 24 hours.
The recommended dosage for Myozyme is 20 mg/kg body weight, administered once every
2 weeks as an intravenous infusion.
Administer infusions incrementally. A starting infusion rate of
1 mg/kg/hour is recommended, with a gradual increase of 2 mg/kg/hour every 30 minutes, in the absence of infusion-associated reactions, up to a maximum rate of 7 mg/kg/hour.